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Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

NCT01784419 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2020-09-29

Study results available
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Summary

This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This new, first of its kind drug is approved for use only in CF patients with the G551D mutation in whom it safely confers considerable benefits. However, it is highly likely that CF patients with many other mutations can benefit similarly from this drug, some of whom can be identified by phenotype or genotype.

We will enroll up to 30 CF subjects with clinical presentations in which there is one or more signs of residual CF channel function. The signs of residual function include: normal digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF disease in a CF patient with severe gene mutations. The primary outcome measure will be the difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor. Secondary outcome measured will be lung function.

Conditions

Interventions

DRUG

ivacaftor

Eligible subjects were randomized to ivacaftor 150 mg by mouth twice a day for 14 days followed by placebo for 14 days, or vice versa. Randomization was based on a computer-generated schedule produced by the research pharmacy, which was concealed from study personnel until study completion. Ivacaftor was purchased at full retail cost and encapsulated with sucrose to match the sucrose-filled placebo capsules. Prior to beginning study drug, there was a 2-week run-in period to ensure clinical stability, assessed by modified Fuchs criteria and pulmonary function. There was a washout period of a minimum of 14 days between study drug cycles to account for carryover effect. The washout period was extended to 6 weeks for subjects on alternating cycles of inhaled antibiotics to coordinate the study drug cycle with the inhaled antibiotic cycle.

DRUG

Placebo

Eligible subjects were randomized to ivacaftor 150 mg by mouth twice a day for 14 days followed by placebo for 14 days, or vice versa. Randomization was based on a computer-generated schedule produced by the research pharmacy, which was concealed from study personnel until study completion. Ivacaftor was purchased at full retail cost and encapsulated with sucrose to match the sucrose-filled placebo capsules. Prior to beginning study drug, there was a 2-week run-in period to ensure clinical stability, assessed by modified Fuchs criteria and pulmonary function. There was a washout period of a minimum of 14 days between study drug cycles to account for carryover effect. The washout period was extended to 6 weeks for subjects on alternating cycles of inhaled antibiotics to coordinate the study drug cycle with the inhaled antibiotic cycle.

Sponsors & Collaborators

Principal Investigators

  • Dennis W Nielson, MD, PhD · University of California, San Francisco

Study Design

Allocation
RANDOMIZED
Purpose
SCREENING
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-10-31
Primary Completion
2015-12-31
Completion
2015-12-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01784419 on ClinicalTrials.gov